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Blood, 1 March 2007, Vol. 109, No. 5, pp. 2256-2262.
Prepublished online as a Blood First Edition Paper on October 12, 2006; DOI 10.1182/blood-2006-07-036657.
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Submitted July 20, 2006
Accepted October 5, 2006
Unrelated donor bone marrow transplantation for the treatment of Fanconi anemia
John E Wagner*, Mary Eapen, Margaret L. MacMillan, Richard E. Harris, Ricardo Pasquini, Farid Boulad, Mei-Jie Zhang, and Arleen D. Auerbach
Division of Pediatric Hematology, University of Minnesota Medical School, Minneapolis, MN
Center for International Blood & Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI
Cincinnati Children's Hospital, Cincinnati, OH
Hospital de Clinicas-Federal University of Parana, Parana, Brazil
Memorial Sloan-Kettering Cancer Center, New York, NY
Department of Hematology and Human Genetics, Rockefeller University, New York, NY
* Corresponding author; email: wagne002{at}umn.edu.
Bone marrow transplantation (BMT) is the only known cure for the hematological manifestations of Fanconi anemia (FA). Potential benefits of unrelated donor BMT for FA, however, have been severely limited by graft rejection and treatment-related mortality with resultant poor survival. Therefore, we evaluated the impact of potential prognostic factors on hematopoietic recovery, graft-versus-host disease (GVHD) and mortality in 98 recipients of unrelated donor BMT, transplanted between 1990 and 2003. Probabilities of neutrophil (89% vs. 69%, p=0.02) and platelet (74% vs. 23%, p<0.001) recovery were higher after fludarabine than non-fludarabine containing regimens. Risks of acute GVHD (RR 4.29, p<0.001) were higher with non T-cell depleted grafts. Day-100 mortality rate was significantly higher after non-fludarabine than fludarabine containing regimens (65% vs. 24%, respectively p<0.001). Corresponding 3-year adjusted overall survival rates were 13% vs. 52% (p<0.001). In addition, mortality was higher in recipients who were older (>10 years), CMV seropositive and received >20-blood product transfusions pre-BMT. Based on these results significant practice changes are suggested: use of a fludarabine containing conditioning regimen in the context of T cell depleted marrow allografts and earlier referral for transplantation prior to excessive transfusions in patients with marrow failure.
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